Exicure Presents Positive Topline Phase 2 Data for Burixafor in Multiple Myeloma at 2025 ASH Annual Meeting
MWN-AI** Summary
Exicure, Inc. (Nasdaq: XCUR), a biotechnology firm focused on hematologic diseases, presented promising topline data from its Phase 2 trial of burixafor (GPC-100) at the 2025 American Society of Hematology (ASH) Annual Meeting. The study explored the use of burixafor, in conjunction with propranolol and granulocyte colony-stimulating factor (G-CSF), for mobilizing hematopoietic progenitor cells (HPCs) in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT). The trial reported that approximately 90% of participants met the key endpoint of mobilizing at least 2 × 10^6 CD34+ cells/kg within two leukapheresis sessions.
In the open-label, multicenter trial involving 19 patients, 17 achieved the primary endpoint, while median times to neutrophil and platelet engraftment post-transplant were recorded at 13 and 17.5 days, respectively. Burixafor’s ability to rapidly mobilize HPCs, with peak levels seen within one hour of administration, stood out as a significant advantage over existing CXCR4 inhibitors, facilitating same-day administration and apheresis procedures.
Remarkably, a majority of participants had prior exposure to daratumumab, a therapy that can hinder mobilization efforts, yet 87.5% of these patients achieved the primary endpoint. The therapeutic regimen was well tolerated, showing no burixafor-related adverse events above Grade 2.
Dr. Jack Khouri, the study's lead investigator, expressed optimism regarding the results, highlighting the treatment's potential to improve patient experiences significantly. In addition to multiple myeloma, burixafor is positioned as a contender for other diseases requiring enhanced cell mobilization, thus broadening its therapeutic horizon. Exicure aims to leverage burixafor’s capabilities not only in hematology but also in supporting cell and gene therapies.
MWN-AI** Analysis
Exicure, Inc. (Nasdaq: XCUR) has recently released promising Phase 2 trial results for their lead candidate, burixafor (GPC-100), demonstrating significant potential in enhancing hematopoietic progenitor cell (HPC) mobilization in multiple myeloma patients. With approximately 90% of participants achieving the trial’s primary endpoint, the results could serve as a pivotal catalyst for both patient outcomes and Exicure's market position.
The unique mechanism of burixafor, as a CXCR4 antagonist, facilitates rapid mobilization of HPCs, a crucial step in autologous hematopoietic cell transplantation (AHCT). This is particularly noteworthy given the inclusion of patients previously treated with daratumumab, a therapy that is known to complicate cell mobilization. The ability of burixafor to deliver rapid and robust mobilization, within a streamlined timeframe of one hour of administration, suggests a transformative approach in patient care.
Investors should watch closely as the data indicate that burixafor could not only enhance the efficiency of AHCT in multiple myeloma but potentially expand its application across various hematologic malignancies, including acute myeloid leukemia and sickle cell disease. Furthermore, the favorable safety profile with no higher than Grade 2 adverse events reinforces the drug's viability.
Economically speaking, these results position Exicure to attract further investments and collaborations, enhancing their development pipeline. The market response may vary based on broader industry trends and competitor activities, but the paradigm-shifting potential demonstrated by this data makes Exicure an attractive proposition for investors focused on biotech sectors.
In conclusion, Exicure stands poised for growth, with a competitive edge from burixafor's differentiated profile. Monitoring subsequent trials and the company’s strategic plans for commercialization will be essential for stakeholders considering entry into or addition to their portfolios in this high-stakes market.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
REDWOOD CITY, Calif., Dec. 08, 2025 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced positive results from its completed Phase 2 trial evaluating burixafor (GPC-100) in combination with propranolol and granulocyte colony-stimulating factor (G-CSF) for the mobilization of hematopoietic progenitor cells (HPCs) in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT). The data, which showed that approximately 90% of study participants achieved the primary endpoint, were presented today in an oral session at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida.
Burixafor is an investigational small molecule that blocks CXCL12 binding to CXCR4 receptors on HPCs, rapidly mobilizing these cells from the bone marrow into the peripheral blood. In preclinical studies, propranolol enhanced burixafor-induced mobilization by inhibiting the ?2-adrenergic receptor (?2AR).
In the open-label, multicenter Phase 2 trial (NCT05561751), 17 of 19 participants (89.5%) achieved the primary endpoint of collecting ?2 × 10? CD34+ cells/kg within two leukapheresis sessions. Two required another session to achieve 2x10 6 CD34+ cells/kg. Among participants who proceeded to transplant, the median time to neutrophil engraftment was 13 days, and the median time to platelet engraftment was 17.5 days. Burixafor has a differentiated and rapid mobilization kinetics, with peak peripheral levels of CD34+ cells observed within one hour of administration. This distinguishes it from other CXCR4 inhibitors and allows for same day burixafor administration and apheresis.
Notably, 16 of 19 participants (84.2%) had prior exposure to daratumumab, a therapy associated with reduced mobilization, yet 14 of those 16 participants (87.5%) achieved the primary endpoint, including 12 of 14 participants (85.7%) who had received both daratumumab and lenalidomide. Longer intervals between the last dose of daratumumab and leukapheresis were associated with higher CD34+ cell yields.
Burixafor administered in combination with propranolol and G-CSF was well tolerated and demonstrated an excellent safety profile. There were no burixafor-related adverse events higher than Grade 2.
“In this Phase 2 study, the combination of burixafor, G-CSF and propranolol showed an excellent safety profile and supported reliable mobilization of hematopoietic progenitor cells, allowing all participants who elected to proceed with transplant to undergo AHCT and successfully engraft,” said Jack Khouri, M.D., Associate Professor of Medicine at the Cleveland Clinic Lerner College of Medicine, Case Western Reserve University and the study’s lead investigator. “We’re encouraged by these results, particularly given the high proportion of participants previously treated with daratumumab, an agent which may reduce stem cell yield. The ability to achieve peak circulating HPC levels rapidly after burixafor administration for immediate leukapheresis also has the potential to meaningfully improve the patient experience by reducing the burden of the mobilization process.”
Oral Presentation Details
Abstract Number: 1050
Title: An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma
Presenter: Dr. Jack Khouri, Associate Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University
Session: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Refining CAR-T Cells and Engineered HSPCs; New Approaches to HSPC mobilization
Date and Time: December 8, 2025, 5:45-6:00pm EST
Location: Hyatt - Regency Ballroom R
About Burixafor (GPC-100)
Burixafor (GPC-100) is a highly selective small molecule antagonist of CXCR4, a chemokine receptor that plays a central role in retaining hematopoietic stem cells in the bone marrow niche. By blocking CXCR4, burixafor may enhance the mobilization of these cells into the peripheral blood for collection and use in autologous stem cell transplant (ASCT) procedures. Originally developed by GPCR Therapeutics, Inc., burixafor became part of Exicure’s pipeline following the company’s acquisition in January 2025. In addition to multiple myeloma, burixafor is also being considered in other diseases where improved stem cell mobilization could help enable more efficient and effective treatment approaches, such as sickle cell disease, rare diseases requiring autologous transplant, and cell and gene therapy settings. A chemosensitization trial in AML is also being planned, leveraging burixafor’s mechanism of mobilizing malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy.
About Exicure
Exicure, Inc. (Nasdaq: XCUR) is a clinical-stage biotechnology company developing therapies to address key challenges in hematologic diseases. The company’s lead program, burixafor (GPC-100), is being evaluated for its ability to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. It is also being studied as a potential chemosensitizing agent in acute myeloid leukemia (AML). For more information, visit www.exicuretx.com .
Media Contact:
Sarah Ellinwood, PhD
Kendall Investor Relations
sellinwood@kendallir.com
FAQ**
How do the positive Phase 2 trial results for Exicure Inc. XCUR's burixafor (GPC-100) impact the company's future prospects in the hematologic disease market, especially in the context of multiple myeloma therapies?
Given the high percentage of participants achieving the primary endpoint in Exicure Inc. XCUR's trial, what strategies will the company employ to ensure successful commercialization of burixafor (GPC-100)?
How might Exicure Inc. XCUR's findings on burixafor’s rapid mobilization of hematopoietic progenitor cells influence treatment protocols in autologous hematopoietic cell transplantation (AHCT)?
In light of Exicure Inc. XCUR’s plans to explore burixafor in additional conditions beyond multiple myeloma, what potential does the company see for this therapy in addressing other hematologic or oncologic diseases?
**MWN-AI FAQ is based on asking OpenAI questions about Exicure Inc. (NASDAQ: XCUR).
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